New gene therapy shows promising results for the treatment of neurodegenerative disorders -
A new gene therapy approach to replace the enzyme that is deficient in patients with inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brain of treated mice, the restored enzyme function, and prolonged survival of about 2.5 times. The implications of these promising results for the development of similar gene therapies for use in hu-mans and to target additional brain disorders are discussed in two articles published in Human Gene Therapy , a peer review Mary Ann Liebert, Inc., publishers. The articles are part of a special issue on disorders of the central nervous system and are available for free download on the Human Gene Therapy site until August 28, 2016.
Both studies demonstrate the feasibility and efficiency of gene transfer in preclinical models. The articles are titled "Novel Vector Design and hexosaminidase Varieant Enable Virus adeno-associated self-complementary for the treatment of Tay-Sachs disease," by Karumuthil-Melethil, et al. and "Gene Transfer of systemic Using a variant of hexosaminidase scAAV9.47 Vector Corrects Gangliosidosis in GM2 Sandhoff mice" by Osmon et al.
Steven Gray, University of North Carolina at Chapel Hill, and Jagdeep Walia, Queen University (Kingston, Canada), led a team of researchers from SickKids and University of Toronto (Canada), the New Hope Research Foundation (North Oaks, MN), and the University of Mannitoba (Winnipeg, Canada), in the successful development of a company specialized adeno-associated virus (AAV) designed to deliver a gene encoding portions alpha and beta subunits of the enzyme that are defective in mice Tay-Sachs and Sandhoff, respectively. The gene transfer vector novel, administered intravenously, was able to deliver the therapeutic gene in the brain and spinal cord, the target site of action.
"This is an important proof of concept study provides important information about the optimal design value of rAAV vectors for this class of disorders," says the editor Terence R. Flotte, MD, Professor Celia and Isaac Haidak of medical education and Dean, Provost and Executive Vice Chancellor, University of Massachusetts medical School, Worcester, MA.
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